U.S. FDA Grants Full Approval of Kite’s Tecartus for Adult Patients with Relapsed or Refractory Mantle Cell Lymphoma

The U.S. Food and Drug Administration (FDA) has granted traditional (full) approval to Kite’s CAR T-cell therapy, Tecartus (brexucabtagene autoleucel), for adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL). The full approval now includes efficacy, safety and pharmacokinetic data from Cohort 3 of the ZUMA-2 study in patients who are R/R after one or more lines of therapy and who are Bruton tyrosine kinase inhibitor (BTKi)-naïve.

Today’s action converts the R/R MCL indication to full approval based on the totality of evidence from ZUMA-2, including confirmatory data from Cohort 3, which demonstrated high response rates, durable efficacy outcomes and a manageable safety profile consistent with prior experience. This milestone fulfills Kite’s post-marketing requirement for verification and description of clinical benefit in a confirmatory trial under the FDA’s Accelerated Approval pathway for Tecartus in R/R MCL.

“The full approval of brexu-cel in relapsed or refractory mantle cell lymphoma, along with the inclusion of Cohort 3 data in the label, reinforces our confidence in the overall profile of brexu-cel,” said Michael Wang, M.D., ZUMA-2 Lead Investigator and Professor, Department of Lymphoma and Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center. “The Cohort 3 results showed high response rates, including deep remissions, in patients who were Bruton tyrosine kinase inhibitor-naïve, with a manageable safety profile consistent with prior experience. These data provide important information to help guide treatment decisions in the relapsed or refractory setting for appropriate patients.”

MCL is a rare form of non-Hodgkin lymphoma (NHL) that arises from cells originating in the “mantle zone” of the lymph node and predominantly affects men over the age of 60. Approximately 33,000 people worldwide are diagnosed with MCL each year. MCL is highly aggressive following relapse, with many patients’ disease progressing following therapy.

“The full approval of Tecartus for adults with relapsed or refractory mantle cell lymphoma reflects meaningful progress,” said Gallia Levy, MD, PhD, Senior Vice President and Global Head of Development, Kite. “For those whose cancer has returned, the data support Tecartus as a second-line treatment option with the potential to deliver long-term remission.”

Data Supporting Full Approval of Tecartus in R/R MCL

ZUMA-2 is a single-arm, open-label, multicenter study evaluating Tecartus in adult patients with R/R MCL. Cohorts 1 and 2 evaluated Tecartus in patients who had previously received up to five lines of therapy including anthracycline- or bendamustine-containing chemotherapy, an anti-CD20 antibody and a BTKi. Cohort 3 evaluated Tecartus in patients who had received up to five prior lines of therapy and were BTKi-naïve. A total of 82 patients were treated in Cohorts 1 and 2, and 86 patients were treated in Cohort 3. The primary endpoint across the study was objective response rate (ORR) per the Lugano Classification (2014), as assessed by an Independent Radiologic Review Committee.

U.S. FDA Grants Full Approval of Kite’s Tecartus for Adult Patients with Relapsed or Refractory Mantle Cell Lymphoma

FDA Approves Revtorpyk (gedatolisib) for the Treatment of HR+/HER2-, PIK3CA Wild-Type Locally Advanced or Metastatic Breast Cancer

Celcuity Inc. (Nasdaq: CELC), a biotechnology company focused on developing and commercializing targeted therapies for multiple solid tumor indications,   announced  the U.S. Food and Drug Administration (“FDA”) approval of  Revtorpyk (gedatolisib) for the treatment of patients with hormone receptor positive (“HR+”), human epidermal growth factor receptor 2 negative (“HER2-”), locally advanced or metastatic breast cancer without a PIK3CA mutation detected following progression on or after treatment with at least one line of endocrine therapy in the metastatic setting. Revtorpyk is the only inhibitor of class I PI3K isoforms (α, β, δ, γ) and mTOR complexes mTORC1 and mTORC2 to receive FDA approval.

• Revtorpyk is the first and only FDA-approved therapy that inhibits all class I PI3K isoforms (α, β, δ, γ) and mTOR complexes mTORC1 and mTORC2
• In the Phase 3 VIKTORIA-1 trial, Revtorpyk combined with palbociclib and fulvestrant and Revtorpyk combined with fulvestrant reduced the risk of disease progression or death by 76% and 67%, respectively, compared to fulvestrant among patients with PIK3CA wild-type advanced or metastatic breast cancer

“The PI3K/AKT/mTOR, or PAM, pathway is one of the most important targets in cancer, but comprehensively inhibiting it has stymied researchers and drug developers for nearly two decades,” said Brian Sullivan, CEO and co-founder of Celcuity. “Revtorpyk addressed this 20-year challenge by becoming the first pan-PI3K, mTORC1/2 inhibitor approved by the FDA. We are thankful for the opportunity to make this important new therapy available to patients with HR+/HER2- locally advanced or metastatic breast cancer.”

HR+/HER2- breast cancer is the most common subtype of breast cancer, accounting for approximately 70% of all breast cancers.1 Among this breast cancer subtype, approximately 60% have PIK3CA wild-type disease.2

“For patients with HR+/HER2- locally advanced or metastatic breast cancer, there is an urgent need for new treatment options that can meaningfully increase the likelihood of survival without disease progression or death,” said Sara Hurvitz, MD, Senior Vice President, Clinical Research Division, Fred Hutchinson Cancer Center, Smith Family Endowed Chair in Women’s Health and Professor and Head, Division of Hematology and Oncology, University of Washington, School of Medicine and co-principal investigator for the VIKTORIA-1 trial. “With the approval of Revtorpyk, oncologists now have an effective new treatment option for these patients.”

The approval of Revtorpyk is based on positive clinical results from the PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 trial, an open-label, global, randomized clinical trial evaluating the efficacy and safety of Revtorpyk plus fulvestrant, with or without palbociclib, for the treatment of patients with locally advanced or metastatic HR+/HER2- breast cancer following progression on or after CDK4/6 therapy and an aromatase inhibitor. In the VIKTORIA-1 trial, median progression free survival (“PFS”) with the Revtorpyk triplet (Revtorpyk plus palbociclib and fulvestrant) was 9.3 months versus 2.0 months with fulvestrant, an incremental improvement of 7.3 months (HR=0.24; 95% CI: 0.17-0.35; p<0.0001). The objective response rate (“ORR”) of the Revtorpyk triplet was 32% compared to 1% with fulvestrant and the median duration of response (“DOR”) was 17.5 months. For the Revtorpyk doublet (Revtorpyk plus fulvestrant), the median PFS was 7.4 months versus 2.0 months with fulvestrant, an incremental improvement of 5.4 months (HR=0.33; 95% CI: 0.24-0.48; p<0.0001). The ORR of the Revtorpyk doublet was 28% and the median DOR was 12.0 months. The median DOR was not determinable for fulvestrant because there was only one objective response.

“Today’s approval of Revtorpyk addresses a significant unmet need for the thousands of patients affected each year by HR+/HER2-, PIK3CA wild-type locally advanced or metastatic breast cancer whose disease has progressed after endocrine therapy,” said Igor Gorbatchevsky, MD, Chief Medical Officer of Celcuity. “We are deeply grateful to the patients and their caregivers, investigators and clinical study teams, and Celcuity team members who made this advancement possible.”

Celcuity anticipates commercial launch in late Q3 2026. Based on the company’s commitment to ensuring broad, affordable, and unrestricted patient access to Revtorpyk, we have designed a comprehensive patient support program. To make Revtorpyk available to patients prior to commercial launch, those who are eligible will be able to enroll in Celcuity’s expanded access program.

Celcuity plans to submit in Q3 2026 a supplemental New Drug Application (“sNDA”) to the FDA for Revtorpyk for the treatment of HR+/HER2-, PIK3CA mutated, locally advanced or metastatic breast cancer, following at least one line of endocrine therapy based on results from the mutant cohort of the Phase 3 VIKTORIA-1 trial. Results for this study were recently presented in a late-breaking abstract oral session at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting. Following the sNDA submission, Celcuity intends to submit VIKTORIA-1 data for marketing authorization of gedatolisib to other regulatory authorities around the world.

Revtorpyk is also being studied in the ongoing Phase 3 VIKTORIA-2 clinical trial incorporating two independent studies evaluating two separate patient cohorts with HR+/HER2- locally advanced or metastatic breast cancer who are treatment-naive in the advanced setting.

About Revtorpyk (gedatolisib)

Revtorpyk is a kinase inhibitor of class I PI3K isoforms (α, β, δ, γ) and mTOR complexes mTORC1 and mTORC2, resulting in downstream inhibition of multiple effectors, including AKT.3.4.5

Revtorpyk is in development for the first-line treatment of HR+/HER2- locally advanced or metastatic breast cancer and for the second-line treatment of metastatic castration resistant prostate cancer.

Indication Statement

Revtorpyk (gedatolisib) is a kinase inhibitor indicated in combination with fulvestrant, with or without palbociclib, for the treatment of adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer without a PIK3CA mutation detected following progression on or after treatment with at least one line of endocrine therapy in the metastatic setting.

https://en.wikipedia.org/wiki/Gedatolisib

FDA Approves Revtorpyk (gedatolisib) for the Treatment of HR+/HER2-, PIK3CA Wild-Type Locally Advanced or Metastatic Breast Cancer

FDA Accepts New Drug Application for Genentech’s Giredestrant in ESR1-Mutated, ER-Positive Advanced Breast Cancer

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced  the U.S. Food and Drug Administration (FDA)  acceptance of  the company’s New Drug Application for giredestrant, an investigational oral therapy, in combination with everolimus for the treatment of adult patients with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2-negative, ESR1-mutated locally advanced or metastatic breast cancer following recurrence or progression on a prior endocrine-based regimen. The FDA is expected to make a decision on the approval by December 18, 2026. Giredestrant plus everolimus could be the first and only oral selective estrogen receptor degrader (SERD) combination approved in the post-cyclin-dependent kinase (CDK)4/6 inhibitor setting.

Filing acceptance based on Phase III data showing giredestrant plus everolimus reduced the risk of disease progression or death by 44% and 62% in ITT and ESR1-mutated populations, respectively, versus standard-of-care endocrine therapy plus everolimus

Strength of evERA data demonstrate potential for giredestrant combination to help address resistance to standard-of-care therapies, and could be the first and only oral SERD combination approved in the post-CDK4/6 inhibitor setting

“The clinically meaningful benefit seen with giredestrant could enable an important new treatment option to help delay disease progression or death in people with advanced, ER-positive breast cancer,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “This acceptance marks a first step towards establishing the giredestrant combination as a new standard of care in this population.”

The filing acceptance is based on the Phase III evERA Breast Cancer study results, which showed that giredestrant plus everolimus reduced the risk of disease progression or death by 44% and 62% in the intention-to-treat (ITT) and ESR1-mutated populations, respectively, compared with standard-of-care endocrine therapy plus everolimus. In the ESR1-mutated population, the median progression-free survival (PFS) was 9.99 months compared with 5.45 months in the giredestrant and comparator arm, respectively (stratified hazard ratio [HR]=0.38, 95% CI: 0.27-0.54, p-value=<0.0001). In the ITT population, the median PFS was 8.77 months compared with 5.49 months in the giredestrant and comparator arms, respectively (HR=0.56, 95% CI: 0.44-0.71, p-value=<0.0001).

Overall survival (OS) data were immature at the time of analysis, but a clear positive trend has been observed in the ITT (HR=0.69, 95% CI: 0.47-1.00, p-value=0.0473) and ESR1-mutated populations (HR=0.62, 95% CI: 0.38-1.02, p-value=0.0566). Follow-up for OS will continue to the next analysis. Adverse events for the giredestrant combination were manageable and consistent with the known safety profiles of the individual medicines. No unexpected safety findings were observed, including no photopsia.

Data from evERA are being used to support filing submissions to other global health authorities.

ER-positive breast cancer accounts for approximately 70% of breast cancer cases. Resistance to endocrine therapies, particularly in the post-CDK inhibitor setting, increases the risk of disease progression and is associated with poor outcomes. Oral combination therapies, such as giredestrant plus everolimus, could address this by targeting two different signaling pathways while helping to minimize the impact of treatment on people’s lives without the need for injections.

evERA was the first positive Phase III readout for giredestrant, followed by lidERA Breast Cancer in the early-stage setting. The scientific rationale for lidERA was supported by prior results in the neoadjuvant setting, including the coopERA trial showing that giredestrant was superior to an aromatase inhibitor in reducing malignant cell division (Ki67 levels). This growing body of evidence underscores the potential of giredestrant to become a new standard-of-care endocrine therapy across ER-positive early-stage and advanced breast cancer. In the coming weeks, Genentech will submit the giredestrant Phase III lidERA data in early-stage breast cancer to the FDA. The persevERA readout in first-line ER-positive breast cancer is expected in the first half of this year, which will provide further evidence for giredestrant in the ER-positive breast cancer treatment paradigm.

Our extensive giredestrant clinical development program spans multiple treatment settings and lines of therapy, reflecting our commitment to deliver innovative medicines to as many people with ER-positive breast cancer as possible.

https://en.wikipedia.org/wiki/Giredestrant

Xspray Pharma Re-Submits its FDA Application for Dasynoc

Xspray Pharma AB (publ) has re-submitted its application for market approval for Dasynoc® to the U.S. Food and Drug Administration (FDA). Dasynoc® is the company's lead product candidate – an amorphous dasatinib for the treatment of leukemia. The resubmission includes the additional information requested by the FDA. A new PDUFA-date is expected to be set for August 2026 at the latest.

Xspray Pharma has submitted its response to the CRL (Complete Response Letter) received from the FDA in October 2025. The CRL related to the company’s NDA application (New Drug Application) for market approval in the United States for Dasynoc, an amorphous dasatinib for the treatment of CML (chronic myeloid leukemia) and ALL (acute lymphoblastic leukemia).

The current resubmission includes additional data specifically designed to address FDA’s concerns regarding risk for medication error. A qualitative human factors study has been completed to address this, confirming high prescriber comprehension using the new tablet strengths. The requested documentation confirming the effectiveness of previously implemented manufacturing measures was also submitted.

The GMP-observations in the company’s third-party manufacturing facility are being handled by the facility itself, and corrective measures are being implemented. Xspray welcomes the recent acquisition of the facility by Benta Group, a well-established company with core operations in pharmaceutical contract manufacturing and a strong reputation for quality, regulatory compliance and operational standards.

“Although the progress and approval process of the facility is not under our direct control, current information that is available to us reassures us that the issues in the facility will be resolved. We maintain our plan to launch two products, Dasynoc and XS003 nilotinib during the second half of 2026,” comments Per Andersson, CEO of Xspray Pharma.

The Dasynoc application will now undergo FDA review. Once the FDA formally confirms acceptance of the application, a new PDUFA date will be announced, expected no later than August 2026.

https://en.wikipedia.org/wiki/Dasatinib

Telix Resubmits NDA to U.S. FDA for TLX101-Px (Pixclara) Brain Cancer Imaging Candidate

Telix Pharmaceuticals Limited (ASX: TLX, NASDAQ: TLX, “Telix”) announced the resubmission of a New Drug Application (NDA) to the United States (U.S.) Food and Drug Administration (FDA) for TLX101-Px, (Pixclara®1, Floretyrosine F 18 or 18F-FET), an investigational PET2 imaging agent for the characterization of recurrent or progressive glioma (brain cancer) from treatment related changes in both adult and pediatric patients.

Telix has resubmitted the NDA with the additional data requested by the FDA. The Company believes, based on the Type A meeting and ongoing consultation with the FDA, that the additional data and statistical analysis, along with the primary data set provided in the original submission, appropriately addresses the Complete Response Letter3.

Given the potential to address significant unmet medical need, TLX101-Px has been granted Orphan Drug4 and Fast Track5 designations by the FDA. PET imaging with 18F-FET is already included in international clinical practice guidelines for the imaging of gliomas6, however there is currently no FDA-approved targeted amino acid PET agent for adult and pediatric brain cancer imaging commercially available in the U.S.

Dr. David N. Cade, Telix Group Chief Medical Officer, said, “We appreciate the FDA’s recognition of the critical unmet need to improve the diagnosis and management of glioma, particularly in the post-treatment setting. Our resubmission is supported by an extensive and compelling data set – particularly so for an orphan indication. We are grateful to our global clinical collaborators, who share our commitment to ensuring patients in the U.S. can benefit from this important patient management tool.”

Maggie Haynes, Executive Director, Head for the Cure Foundation, added: “Our community is encouraged by the FDA’s ongoing engagement and guidance to the sponsor and support for the Expanded Access Program for TLX101-Px. We are hopeful of an expedited review, so this important and proven imaging option can become available to those who urgently need it.”

https://en.wikipedia.org/wiki/Fluoroethyl-L-tyrosine_(18F)

Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC

Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today announced the submission to the U.S. Food and Drug Administration (FDA) of the Company's NDA for neladalkib, an investigational ALK-selective inhibitor, in TKI pre-treated advanced ALK-positive NSCLC.

New Drug Application (NDA) based on data in TKI pre-treated patients from the global ALKOVE-1 Phase 1/2 clinical trial

"The advancement of neladalkib from first clinical trial initiation to NDA submission in less than four years represents a remarkable pace in oncology drug development, underscoring the vigor and urgency our team brought to this program and our deep commitment to the ALK-positive NSCLC community," said Darlene Noci, A.L.M., Chief Development Officer at Nuvalent. "We would like to extend our sincere gratitude to the patients, families and investigators who have made this progress possible, and are committed to working closely with the FDA throughout the NDA review process toward our goal of bringing neladalkib to patients as quickly as possible."

The application is based on data in TKI pre-treated patients with advanced ALK-positive NSCLC treated with neladalkib in the global, registration-directed ALKOVE-1 Phase 1/2 clinical trial. In this population, neladalkib demonstrated encouraging overall activity, including intracranial responses, the ability to address key drivers of disease progression, and a generally well-tolerated safety profile consistent with its ALK-selective, TRK-sparing design. The company plans to share detailed results at a future medical meeting.

Neladalkib has received breakthrough therapy designation from the FDA for the treatment of patients with locally advanced or metastatic ALK-positive NSCLC who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

About Neladalkib

Neladalkib is an investigational, brain-penetrant, ALK-selective inhibitor created with the aim to overcome limitations observed with currently available ALK inhibitors. Neladalkib is designed to remain active in tumors that have developed resistance to first-, second-, and third-generation ALK inhibitors, including tumors with single or compound treatment-emergent ALK mutations such as G1202R. In addition, neladalkib is designed for central nervous system (CNS) penetrance to improve treatment options for patients with brain metastases, and to avoid inhibition of the structurally related tropomyosin receptor kinase (TRK) family. Together, these characteristics have the potential to avoid TRK-related CNS adverse events seen with dual TRK/ALK inhibitors and to drive deep, durable responses for patients across all lines of therapy. Neladalkib has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with locally advanced or metastatic ALK-positive non-small cell lung cancer (NSCLC) who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

https://go.drugbank.com/drugs/DB16688

Aldeyra Therapeutics Receives Complete Response Letter from the U.S. Food and Drug Administration for the Reproxalap New Drug Application for the Treatment of Signs and Symptoms of Dry Eye Disease

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to discovering and developing innovative therapies designed to treat immune-mediated diseases, announced receipt of a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for the New Drug Application (NDA) of reproxalap, an investigational drug candidate, for the treatment of dry eye disease. The CRL stated that there is “a lack of substantial evidence consisting of adequate and well-controlled investigations … that the drug product will have the effect it purports or is represented to have under the conditions of use prescribed, recommended, or suggested in its proposed labeling” and that “the application has failed to demonstrate efficacy in adequate and well controlled studies in the treatment of signs and symptoms of dry eye disease.” The letter also stated that the “inconsistency of study results raises serious concerns about the reliability and meaningfulness of the positive findings” and that the “totality of evidence from the completed clinical trials does not support the effectiveness of the product.” Consistent with prior NDA reviews of reproxalap, no safety or manufacturing concerns were identified.

During the NDA review, label drafts were provided by the FDA in December 2025 and again in March 2026. Aldeyra does not believe that label negotiations were completed.

The FDA recommended that the reasons for failure in certain trials be explored, and that populations or certain conditions in which reproxalap may be effective be identified. The FDA did not recommend conducting additional trials or request submission of additional confirmatory evidence. As such, Aldeyra does not currently expect to pursue additional clinical trials, and intends to expeditiously request a Type A meeting to understand the actions needed for NDA approval. Per Prescription Drug User Fee Act (PDUFA) goals, the target Type A meeting date is within 30 days of receipt of the meeting request.

“To the thousands of American and Canadian patients who participated in our clinical trials and to the tens of millions of patients with dry eye disease worldwide, I want to assure you that we will work with urgency to support the FDA in enabling market access to what is, to our knowledge, the only drug with clinical activity within minutes of administration in patients with dry eye disease, a condition that is today treated with medications that require weeks or months of treatment to achieve even modest improvement,” stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra.

https://go.drugbank.com/drugs/DB16688

Cogent Biosciences Announces Submission of New Drug Application for Bezuclastinib in Gastrointestinal Stromal Tumors (GIST)

Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, announced the completion of the submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bezuclastinib in patients with Gastrointestinal Stromal Tumors (GIST) who have received prior treatment with imatinib. Based on the positive results from the PEAK trial, the bezuclastinib NDA was submitted under the FDA’s Real-Time Oncology Review (RTOR) program, which is intended to enable a more streamlined review process. Bezuclastinib was also granted Breakthrough Therapy Designation as a treatment for GIST earlier this year.

Bezuclastinib NDA submitted under the FDA’s RTOR program based on positive results from Phase 3 PEAK trial; bezuclastinib previously granted Breakthrough Therapy Designation in GIST

Bezuclastinib combination demonstrated 16.5 month mPFS and 46% ORR in imatinib-resistant GIST patients, dramatically improving upon the current standard of care

“We are excited to complete our PEAK NDA submission which marks a significant step toward bringing a new therapy to patients with second-line GIST,” said Andrew Robbins, President and Chief Executive Officer. “Based on the strength of the PEAK data, we believe the bezuclastinib combination has the potential to meaningfully change the treatment landscape for these patients. We are grateful to the patients, investigators, and study teams who made this possible.”

Pivotal data from PEAK, a global, randomized Phase 3 clinical trial evaluating bezuclastinib in combination with sunitinib vs. sunitinib monotherapy in patients with GIST who have received prior treatment with imatinib, were reported in November 2025. As disclosed in the top-line results, the bezuclastinib combination demonstrated a substantial and highly statistically significant clinical benefit on the primary endpoint of progression free survival (PFS), reducing risk of disease progression or death compared to the current standard of care by 50% (hazard ratio of 0.50, 95% CI: 0.39 – 0.65). mPFS, as assessed by blinded independent central review, was 16.5 months for the bezuclastinib combination vs. 9.2 months for sunitinib monotherapy. Additionally, the bezuclastinib combination demonstrated an unprecedented ORR in imatinib-resistant patients, with 46% of patients treated with the bezuclastinib combination achieving an objective response compared to 26% of patients treated with sunitinib. The bezuclastinib combination was generally well tolerated, and no unique risks were observed with the novel combination when compared to the known safety profile of sunitinib. Data for overall survival remains immature.

At the time of data cutoff, based on the number of ongoing patients receiving treatment on the bezuclastinib combination arm, the estimated mean duration of treatment for the bezuclastinib combination is projected to exceed 19 months.

Cogent plans to present full results from the PEAK trial at a major medical meeting during the first half of 2026. Additionally, Cogent is on track this quarter to initiate a Phase 2 trial investigating the benefit of the bezuclastinib plus sunitinib combination for first-line GIST patients with exon 9 mutations who are naive to, or recently initiated treatment with, imatinib. The NDA submission with bezuclastinib in Advanced Systemic Mastocytosis (AdvSM) also remains on track for the first half of 2026.

Bezuclastinib - Expanded Access Program

Working with the FDA, Cogent has established active Expanded Access Programs (EAPs) for U.S. patients with GIST or SM who meet disease-specific criteria and could benefit from treatment with bezuclastinib or the combination of bezuclastinib and sunitinib. A growing number of sites now offer access to the bezuclastinib EAPs. For more information please visit: https://www.cogentbio.com/bezuclastinib-program-development/#our-expanded-access-policy

Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Cogent also announced today that, on March 30, 2026, the Compensation Committee of Cogent’s Board of Directors, made up entirely of independent directors, approved the grants of “inducement” equity awards to six new employees under the company’s 2020 Inducement Plan with a grant date of March 30, 2026. The awards were approved in accordance with Listing Rule 5635(c)(4) of the corporate governance rules of the Nasdaq Stock Market. The employees received, in the aggregate, (i) nonqualified options to purchase 21,100 shares of Cogent common stock and (ii) 15,700 restricted stock units (RSUs). Each option has a 10-year term, an exercise price equal to the closing price of Cogent’s common stock on the grant date, and a 4-year vesting schedule with 25% vesting on the 1-year anniversary of the grant date and the remainder vesting in equal monthly installments over the subsequent 36 months, provided such employee remains employed through each such vesting date. The RSUs vest annually in equal installments over 4 years from the grant date, provided such employee remains employed through each such vesting date.

https://en.wikipedia.org/wiki/Bezuclastinib

Nanomedicine offers targeted solutions for breast cancer treatment

Breast cancer (BCA) is one of the most common cancers worldwide, with high mortality and morbidity in women. This review focuses on the applications of nanotechnology, nanomaterials (NMs), and nanoparticles (NPs) in BCA diagnosis and therapy. Nanotechnologies, nanocarriers, and nano-encapsulation versus conventional counterparts are discussed. Various drug formulations into lipid NPs, nanoemulsions, polymeric NPs, and metal-based NPs enhance bioavailability and therapeutic efficacy, overcoming limitations of conventional formulations. Clinical specialists have achieved improved outcomes in BCA detection and monitoring using nanotechnology, ultimately improving patients' quality of life.

Introduction

Cancer is a leading cause of death globally. Breast cancer accounts for 30% of all cancer cases and 15% of cancer‑related deaths in women. The PI3K/AKT/mTOR signaling pathway plays a crucial role in BCA development and progression. Nanomedicine applies NMs and NPs for prevention, diagnosis, and treatment. This review addresses challenges of conventional therapies (lack of target specificity, drug resistance, systemic toxicity) and highlights how nanotechnology overcomes these limitations.

General Aspects of NMs and NPs

NMs have at least one dimension in 1–100 nm and a large surface‑to‑volume ratio, conferring novel properties. Reducing particle size increases solubility and surface interactions. Nanotechnology improves pharmacokinetics, enables targeted delivery, enhances permeability and retention effects in tumors, and reduces required drug doses.

Different Aspects of BCA

Statistics: GLOBOCAN 2022 reported 2,296,840 new BCA cases (ASIR 46.8 per 10⁵) and 666,103 deaths (ASMR 12.7 per 10⁵) worldwide.

Molecular Subtypes: BCA is classified by hormone receptor and HER2 status: Luminal A (~40%, ER⁺/PR⁺, HER2⁻), Luminal B (~20%, ER⁺/PR⁺, HER2⁺/⁻), HER2‑enriched (~10‑15%, ER⁻/PR⁻, HER2⁺), and triple‑negative breast cancer (TNBCA, ~15‑20%, ER⁻/PR⁻/HER2⁻). TNBCA is aggressive, occurs in younger women, has high recurrence and metastasis rates, and lacks targetable proteins.

Challenges: Treatment resistance, recurrence, adverse effects, low cellular absorption, and multidrug resistance.

Therapies: Surgery, chemotherapy, radiotherapy, hormonal therapy, and immunotherapy. Nanotechnology offers innovative carriers to overcome limitations.

Carriers and Nanocarriers for Drug Delivery

Conventional carriers have limited tumor response and affect normal cells. Nanocarriers include lipid nanoparticles (LNPs), nanoemulsions (NEs), polymeric NMs, and metallic NPs. They enhance drug stability, absorption, encapsulation efficiency, bioavailability, and controlled release. NEs improve oral delivery of poorly soluble drugs and reduce toxicity.

Nanocarriers for BCA

Chitosan‑based nanocarriers exploit electrostatic interactions with cancer cells, enhance cellular uptake, and open tight junctions. Quaternary ammonium chitosan improves penetration. Chitosan NPs deliver genes, drugs, and natural compounds; induce phototherapy‑mediated tumor ablation; and support combination therapy.

Clinical results: Nanocarriers improved drug delivery and outcomes. Photothermal nanomaterials (PTT) with nanotechnology enhanced metastatic BCA treatment, reduced damage to healthy cells, and synergized with chemotherapy/immunotherapy. Cyclophosphamide NEs in rats showed remarkable tumor reduction. Exemestane‑loaded polymer‑lipid hybrid nanoparticles (PLH NPs) improved oral bioavailability (>3.5‑fold) and tumor inhibition (62% vs. 31% for conventional suspension) in mice.

Major Metallic Nanocarriers

• Gold (Au) NPs: Biocompatible, easy surface modification, effective against TNBCA via Rad6 conjugation inducing mitochondrial dysfunction. Clinical translation limited by toxicity in liver, kidneys, spleen.

• Silver (Ag) NPs: High photon attenuation; ethyl cellulose‑coated Ag NPs inhibited TNF‑α in BCA cells.

• Copper (Cu) NPs: Bioactive; 5‑fluorouracil loaded into β‑cyclodextrin‑Cu NPs showed sustained release and anticancer activity against TNBCA.

• Iron oxide (Fe₃O₄) NPs: Magnetic core‑shell NPs (Fe₃O₄‑poly(N‑isopropylacrylamide)‑grafted chitosan) delivered methotrexate with 94% entrapment efficiency; enhanced antitumor activity against MCF‑7 cells at 40°C and pH 5.5.

REF: https://www.xiahepublishing.com/2996-3427/OnA-2025-00027

Metformin mimics exercise-related metabolic effects in prostate cancer patients

In continuation of my update on  metformin

A new study has found that metformin, a widely prescribed diabetes drug, may mimic one of exercise's core biological effects in men with prostate cancer, raising levels of a molecule tied to energy balance and weight control even when patients are inactive. The findings suggest metformin could help counter the metabolic strain of hormone therapy, when fatigue and other side effects often limit physical activity.

Led by physician-scientists at Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine, the study appears in the journal EMBO Molecular Medicine.

Exercise is one of the most reliable ways to support health during cancer treatment. It helps regulate weight, blood sugar and cardiovascular health-factors that shape how patients feel during therapy and how well they recover afterward.

For many people with cancer, however, regular exercise isn't always feasible. Fatigue, hormone therapy, pain or advanced disease can limit physical activity precisely when metabolic health becomes most important.

That reality has led researchers to ask a practical question: if exercise confers its benefits through specific biological signals, could some of those signals be activated in other ways?

According to the research, the answer may be yes. Sylvester investigators report that metformin raises levels of a naturally occurring molecule involved in how the body manages energy and weight in prostate cancer patients.

The finding does not suggest that a pill can replace physical activity. Instead, it offers insight into the internal pathways that underlie exercise's metabolic benefits-and how those pathways might still be engaged when movement is limited.

This study reflects what's possible when laboratory science, metabolic biology and clinical investigation are intentionally brought together for transdisciplinary studies. By working across Sylvester's Tumor Biology, Cancer Epigenetics and Translational & Clinical Oncology programs, we were able to link a well-defined molecular signal to real patient data. The result isn't a new cancer biomarker, but a clearer understanding of how a widely used drug may support metabolic health during prostate cancer treatment-an outcome that matters to patients and clinicians alike."

Marijo Bilusic, M.D., Ph.D., Sylvester researcher and first author, genitourinary medical oncologist and professor of medicine and medical oncology at the Miller School.

 At the center of the collaborative, team-science study is a molecule called N-lactoyl-phenylalanine, or Lac-Phe. While its name is technical, its role is relatively simple.

Lac‑Phe is produced when the body is under metabolic demand. It forms when lactate-a substance that accumulates during exertion-combines with phenylalanine, a basic building block of protein. Scientists first took notice of Lac‑Phe because its levels spike after intense exercise, coinciding with shifts in energy use and appetite regulation.

https://en.wikipedia.org/wiki/Metformin

Metformin mimics exercise-related metabolic effects in prostate cancer patients

FDA Approves Komzifti (ziftomenib) for the Treatment of Adults with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

Kura Oncology, Inc. (Nasdaq: KURA) and Kyowa Kirin Co., Ltd. (TSE: 4151)  announced the U.S. Food and Drug Administration (FDA)   approval of Komzifti™ (ziftomenib) for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options. Komzifti is the first and only once-daily, oral menin inhibitor approved for R/R NPM1-mutated (NPM1-m) AML, a devastating blood cancer with limited treatment options.

“Komzifti combines compelling efficacy, a favorable safety profile, compatibility with concomitant medications, and convenient once-daily oral administration in a population with few effective treatment options. These features highlight Komzifti’s potential to serve as the menin inhibitor of choice in its approved indication,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “Together with our partner, Kyowa Kirin, we remain committed to advancing development of Komzifti across the treatment continuum for AML, where its best-in-class profile offers potential for even greater impact in combination regimens and earlier lines of therapy. We are fully prepared to launch Komzifti today and deliver this new medicine to patients in need.”

NPM1 mutations are among the most common founder mutations in AML, occurring in approximately 30% of cases. Historically, approximately 20% of patients with NPM1-m AML do not respond to front-line therapy. Of those who do respond, 70% will relapse within 3 years, most within 12 months. Early relapse and declining survival with each recurrence underscore the urgent need for treatment approaches that deliver lasting remission.

Approval is supported by the pivotal KOMET-001 trial (NCT04067336), which evaluated Komzifti’s safety and efficacy in 112 R/R NPM1-m AML patients. The rate of complete remission (CR) plus CR with partial hematologic recovery (CRh) was 21.4% (95% CI: 14.2, 30.2). The median duration of CR+CRh was 5.0 months (95% CI: 1.9, 8.1) and the median time to first response in patients who achieved a CR or CRh was 2.7 months (range: 0.9 to 15 months). 88% of patients who achieved CR or CRh did so within 6 months of initiating Komzifti. These data from the Prescribing Information are generally consistent with findings recently published in the Journal of Clinical Oncology.1

“Komzifti addresses a critical need for adult patients with R/R NPM1-m AML, many of whom are older and unable to tolerate intensive chemotherapy or transplant,” said Eunice Wang, M.D., Chief of the Leukemia Service and Professor of Oncology at Roswell Park Comprehensive Cancer Center. “The clinical data demonstrate deep and durable responses with a manageable safety profile, including no drug-drug interactions and no Boxed Warnings for QTc prolongation or Torsades de Pointes – key advantages for patients on multiple concurrent medications. This approval equips physicians with a new oral therapy to integrate into care and improve outcomes for this vulnerable patient population.”

The most common adverse reactions (≥20%), including laboratory abnormalities, were aspartate aminotransferase increased, infection without an identified pathogen, potassium decreased, albumin decreased, alanine aminotransferase increased, sodium decreased, creatinine increased, alkaline phosphatase increased, hemorrhage, diarrhea, nausea, fatigue, edema, bacterial infection, musculoskeletal pain, bilirubin increased, potassium increased, differentiation syndrome, pruritus, febrile neutropenia, and transaminases increased. Komzifti includes a Boxed Warning for differentiation syndrome, a well-studied mechanism-based risk in drugs that restore differentiation. Absence of clinically meaningful drug-drug interactions can ease the use of Komzifti with concomitant therapies, including those that cause QTc interval prolongation. QTc interval prolongation was ≤ Grade 3 in 12% of patients and no Grade 4 or Grade 5 QTc interval prolongation was reported. QTc interval prolongation of any cause occurred in 10% of the 70 patients 65 years of age or older.

“The approval of Komzifti underscores our commitment to advancing precision medicines to address the genetic drivers of disease in hematology and oncology,” said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. “In AML, where many patients face severe disease progression and limited treatment options, the evolution toward targeted therapies such as Komzifti represents a major step forward and offers potential to transform existing standards of care. We are proud to partner with Kura Oncology in bringing this important therapy to patients and their families.”

In November 2024, Kura Oncology and Kyowa Kirin entered into a global strategic collaboration to develop and commercialize Komzifti. The collaboration builds on Kyowa Kirin’s leadership and expertise in hematologic malignancies. Under the terms of the collaboration, Kura leads development, regulatory and commercial strategy in the U.S. and is responsible for manufacturing Komzifti. The companies will jointly perform certain commercialization activities in accordance with a co-created U.S. territory commercialization plan. Outside the U.S., Kyowa Kirin leads development, regulatory and commercial strategy and is responsible for commercializing Komzifti.

As part of its commitment to helping patients access Komzifti, Kura has established a support program, Kura RxKonnect™, to minimize barriers to access and reimbursement for appropriate patients prescribed Komzifti. Kura RxKonnect is available 8:00 am – 8:00 pm ET by phone at (844) KuraPSP (844-587-2777) or online at www.KuraRxKonnect.com.

https://en.wikipedia.org/wiki/Ziftomenib

FDA Approves Komzifti (ziftomenib) for the Treatment of Adults with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

FDA Grants Accelerated Approval to Hyrnuo (sevabertinib) for Non-Squamous Non-Small Cell Lung Cancer

Following Priority Review and Breakthrough Therapy Designation, the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Hyrnuo (sevabertinib), for the treatment of adult patients with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have human epidermal growth factor receptor 2 (HER2) tyrosine kinase domain activating mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy. Sevabertinib is an oral, reversible, small molecule, tyrosine kinase inhibitor (TKI).

“The FDA’s approval of sevabertinib elevates the standard of care and provides a new treatment option for patients living with HER2-mutant NSCLC, which is a challenging disease with limited therapies available,” said SOHO-01 lead trialist, Xiuning Le, MD, PhD, The University of Texas MD Anderson Cancer Center, Houston, Texas. “Clinical results from the SOHO-01 trial showed that sevabertinib is effective with a manageable safety profile.”

The FDA approved this indication for sevabertinib under accelerated approval based on objective response rate (ORR) and duration of response (DOR) data from the ongoing Phase I/II SOHO-01 trial (NCT05099172) in patients with advanced NSCLC harboring a HER2-activating mutation, with disease progression after ≥1 systemic therapies for advanced disease. Continued approval of sevabertinib for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Results from the Phase I/II SOHO-01 trial demonstrated for patients naïve to HER2 targeted therapy an ORR of 71% (95% CI: 59%-82%, N=70); 2.9% of patients had a complete response and 69% of patients had a partial response. Median DOR was 9.2 months (95% CI, 6.3–15.0, N=50). Sevabertinib demonstrated a manageable safety profile with a 3.7% discontinuation rate. In the pooled safety population, the most common (> 20%) adverse reactions were diarrhea (87%), rash (66%), paronychia (33%), stomatitis (29%) and nausea (21%).

Positive results from the Phase I/II SOHO-01 trial were presented at the European Society for Medical Oncology (ESMO) Congress 2025 and simultaneously published in the New England Journal of Medicine.0F[1]

“Globally, it is estimated that up to 84,000 people are diagnosed with NSCLC harboring a HER2 mutation each year,” said Christian Rommel, Ph.D., Head of Research and Development at Bayer’s Pharmaceuticals Division. “At Bayer our oncology R&D strategy is centered on precision drug development which is designed to help us address critical unmet medical needs through the advancement of innovative therapies. Today's FDA approval of sevabertinib exemplifies this approach, delivering a targeted treatment specifically for patients with advanced HER2-mutant NSCLC. This significant milestone underscores our unwavering commitment to transforming cancer care by delivering meaningful therapies that have the potential to improve outcomes and extend survival for people living with cancer.”

In 2024, both the U.S. FDA and the CDE in China granted sevabertinib Breakthrough Therapy designation indicating that the compound has the potential to provide substantial improvement over existing therapies in NSCLC with HER2-activating mutations. China accepted a new drug application (NDA) for sevabertinib in July 2025.

https://en.wikipedia.org/wiki/Sevabertinib

FDA Grants Accelerated Approval to Hyrnuo (sevabertinib) for Non-Squamous Non-Small Cell Lung Cancer

FDA Approves Cardamyst (etripamil) Nasal Spray for Adults with Paroxysmal Supraventricular Tachycardia

Milestone Pharmaceuticals Inc. (Nasdaq: MIST) announced  the U.S. Food and Drug Administration (FDA) approved its first commercial product, Cardamyst (etripamil) nasal spray, a prescription medication for the conversion of acute symptomatic episodes of paroxysmal supraventricular tachycardia (PSVT) to sinus rhythm in adults. This approval marks the first time that more than two million Americans with PSVT will have a rapid-acting treatment option they can self-administer outside the emergency department or other healthcare setting. Cardamyst is expected to be available in retail pharmacies in the first quarter of 2026.

Cardamyst nasal spray is a novel and rapid-acting calcium channel blocker delivered when needed to treat often highly symptomatic and unpredictable episodes of PSVT. With Cardamyst, adults with PSVT can be prepared wherever and whenever episodes occur, providing them with active management and a greater sense of control of their condition.

“Cardamyst is a novel at-the-ready treatment option that addresses the unpredictable impact of PSVT by offering patients the freedom to manage episodes anytime and anywhere,” said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. “The FDA approval of Cardamyst is a watershed moment for Milestone and a gratifying event for our team members, patients, clinical investigators, and health care providers who participated in the development program, all of whom I sincerely thank for their dedication, counsel, and collaboration toward this important achievement.”

“Some people with PSVT have endured years of anxiety, fearing their next episode and the stress and disruption of emergency department visits,” said James Ip, M.D., FACC, FHRS, an etripamil investigator. “Cardamyst will give many of them the ability to administer a medication themselves that can quickly stop their PSVT episode and potentially avoid a hospital trip or a call to emergency services.”

Cardamyst Clinical Data

The FDA approval of Cardamyst is supported by a robust clinical trial program based on safety data from more than 1,800 participants and more than 2,000 episodes of PSVT. This includes the successful Phase 3 RAPID trial, a global, randomized, double-blind comparison of Cardamyst vs. placebo, published in The Lancet in 2023. In clinical studies, participants using Cardamyst were two times more likely to convert symptomatic PSVT to sinus rhythm and did so more than three times faster compared with placebo. The RAPID trial achieved its primary endpoint with 64% of those who self-administered Cardamyst (N=99) converting from supraventricular tachycardia (SVT) to sinus rhythm within 30 minutes compared to 31% on placebo (N=85) (HR = 2.62; p<0.001). At one hour, the benefit was demonstrated in 73% of participants. In addition, significant reductions in time to conversion in those who took Cardamyst were evident early and durable, with a median time to conversion of 17 minutes (95% CI: 13.4, 26.5) for those treated with Cardamyst vs. 54 minutes (95% CI: 38.7, 87.3) for those treated with placebo.

A consistent safety profile and treatment effects were observed across all subgroups, including participants concurrently on beta blockers or calcium channel blockers. The most frequent adverse events occurring in ≥5% of participants in randomized clinical trials were mild-to-moderate and transient in nature, including local-site nasal discomfort, nasal congestion, rhinorrhea, throat irritation, and epistaxis. Less than 2% of trial participants discontinued therapy due to adverse events.

For more safety information about Cardamyst, please see Important Safety Information below. For the latest information about the product availability, please see https://milestonepharma.com/.

PSVT, also called SVT, is characterized by episodes of abnormally fast heart rate. Most people with PSVT experience multiple sustained episodes that require treatment on an annual basis. Until now, successful treatment options typically required IV administration in healthcare settings, creating stress and costs for patients and their insurers. Cardamyst offers a new approach to treat episodes of PSVT, enabling adults to self-administer the medication at the onset of symptoms. For more information, please visit SVTHearttoHeart.com.

“Our goal is that Cardamyst will become a trusted and essential solution for healthcare providers and their patients,” said Lorenz Muller, Chief Commercial Officer of Milestone. “Our team is focused on making Cardamyst available to adults with PSVT as quickly as possible, including actively working to secure insurance coverage and begin the distribution of the product through retail pharmacies.”

Annette Greene, Cardamyst clinical trial participant and administrator of the Supraventricular Tachycardia Group on Facebook, with more than 30,000 members, said, “Adults with SVT have been waiting a long time for the day when they can confidently self-administer Cardamyst to treat their SVT episodes. It is very exciting that the day has become a reality.”

Regarding future R&D, Milestone is poised to enter a Phase 3 program in atrial fibrillation with rapid ventricular rate (AFib-RVR), particularly on the strength of the successful ReVeRA Phase 2 trial in AFib-RVR, results of which were published in Circulation: Arrhythmia and Electrophysiology. Incorporating FDA’s guidance, Milestone has developed a Phase 3 registrational program to evaluate self-administered etripamil as a potential treatment for patients with AFib-RVR. When completed, the Company will be following a supplemental New Drug Application (sNDA) regulatory approval pathway for a potential second indication for etripamil in AFib-RVR. As such, Milestone will leverage the approved PSVT indication and the PSVT program data, along with a single pivotal Phase 3 study in patients with AFib-RVR.

https://en.wikipedia.org/wiki/Etripamil

FDA Approves Cardamyst (etripamil) Nasal Spray for Adults with Paroxysmal Supraventricular Tachycardia

FDA Approves Nuzolvence (zoliflodacin) for the Treatment of Uncomplicated Urogenital Gonorrhea in Adults and Adolescents

Innoviva Specialty Therapeutics, a subsidiary of Innoviva, Inc. (NASDAQ: INVA),   announced   the U.S. Food and Drug Administration (FDA)  approval of Nuzolvence (zoliflodacin) for oral suspension, a first-in-class, single-dose oral medication for the treatment of uncomplicated urogenital gonorrhea in adults and pediatric patients 12 years and older weighing at least 35 kg. The development of Nuzolvence was part of a private, not-for-profit collaboration with The Global Antibiotic Research and Development Partnership (GARDP), which sponsored and led the Phase 3 clinical trial that supported FDA approval.

“The FDA’s approval of Nuzolvence marks a pivotal moment for patients and the broader healthcare community managing gonorrhea infections. For the first time in decades, both patients and their healthcare providers will have a single-dose, oral treatment option for uncomplicated urogenital gonorrhea,” said David Altarac, M.D., Chief Medical Officer, Innoviva Specialty Therapeutics. “This achievement underscores our commitment to advancing innovative therapies for infectious diseases and fighting antimicrobial resistance. It highlights the strength of our development capabilities, as well as our collaboration with GARDP and the global scientific community.”

Gonorrhea is the second most common sexually transmitted bacterial infection worldwide, with more than 82 million new cases each year. In the United States alone, the Centers for Disease Control and Prevention (CDC) estimates that over 543,000 cases are reported each year and over 1 million incident cases occur annually,3 underscoring the significant public health impact. The highest rates are seen among sexually active men ages 20-24, though anyone who has unprotected sex can be at risk.4 Without timely treatment, gonorrhea can lead to serious and potentially permanent health complications.

"The decades-long absence of new gonorrhea treatments, combined with rising global antibiotic resistance, has created significant challenges in managing this common but potentially serious sexually transmitted infection," said Edward W. Hook III, M.D., Professor Emeritus of Medicine, University of Alabama at Birmingham, and lead investigator of the Phase 3 Nuzolvence trial. “In the pivotal Phase 3 study, Nuzolvence demonstrated non-inferiority compared to the current standard injectable therapy, including in infections caused by drug-resistant strains, while offering the convenience of a single oral dose.”

“A new antibiotic that does not require injection and can be used for patients who are allergic to penicillin or related drugs meets two important unmet needs in the treatment of gonorrhea,” added Dr. Hook.

The emergence and spread of global drug-resistant infections have led the World Health Organization (WHO) to identify antimicrobial resistance as one of the 10 most critical global health threats.5 The bacterium Neisseria gonorrhoeae has developed resistance to most classes of antibiotics used to treat these infections, including cephalosporins such as ceftriaxone, an injectable treatment, which is the currently recommended first line therapy for uncomplicated urogenital gonorrhea.

The Company plans to commercialize Nuzolvence in the second half of 2026, either in collaboration with a commercialization partner or independently.

https://en.wikipedia.org/wiki/Zoliflodacin

FDA Approves Nuzolvence (zoliflodacin) for the Treatment of Uncomplicated Urogenital Gonorrhea in Adults and Adolescents

FDA Approves Myqorzo (aficamten) for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy

Cytokinetics, Incorporated (Nasdaq: CYTK) announced the U.S. Food and Drug Administration (FDA) approval of  Myqorzo™ (aficamten, 5 mg, 10 mg, 15 mg, 20 mg tablets for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. Myqorzo is an allosteric and reversible inhibitor of cardiac myosin motor activity. In patients with oHCM, myosin inhibition with Myqorzo reduces cardiac contractility and left ventricular outflow tract (LVOT) obstruction.

“This is a historic moment for our company and for the patients we serve, as we fulfill our promise to translate our science into medicines that may make a meaningful difference in patients’ lives,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “Our first FDA approval stands as a testament to the strength of our science and the bold, trailblazing research that has defined Cytokinetics’ leadership in muscle biology. I’m pleased that the approved label and REMS reflect the distinct characteristics of Myqorzo including a straightforward, flexible dosing regimen, no requirement for drug-drug interaction monitoring and a predictable safety profile. I am profoundly grateful for the many years of passion and persistence shown by patients with obstructive HCM, as well as healthcare professionals, advocates, partners and employees who have contributed so importantly to reaching this key milestone.”

The full U.S. Prescribing Information for Myqorzo includes a Boxed WARNING for the risk of heart failure. Myqorzo reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction. Echocardiogram assessments are required prior to and during treatment with Myqorzo to monitor for systolic dysfunction. Initiation of Myqorzo in patients LVEF <55% is not recommended. Decrease the dose of Myqorzo if LVEF <50% and ≥40%. Interrupt the dose of Myqorzo if LVEF <40% or if the patient experiences heart failure symptoms or worsening clinical status due to systolic dysfunction. Because of the risk of heart failure due to systolic dysfunction, Myqorzo is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Myqorzo REMS Program. Please see additional Important Safety Information including Boxed WARNING below.

Myqorzo is expected to be available in the U.S. in the second half of January 2026. Cytokinetics will support patients with Myqorzo & You™, a personalized program for patients prescribed Myqorzo in the U.S. to help navigate the treatment journey, provide disease and product education, and offer support with insurance benefits investigations or financial assistance for those eligible. For more information, call 833-Myqorzo (833-697-6796).

“HCM is a heart muscle disease associated with a significant symptom burden. This approval of a new drug, Myqorzo, represents a meaningful addition to the treatment options available for symptomatic obstructive HCM patients,” said Martin Maron, M.D., Director, Hypertrophic Cardiomyopathy Center, Lahey Hospital and Medical Center, and Principal Investigator of SEQUOIA-HCM. “In SEQUOIA-HCM, Myqorzo improved exercise capacity and reduced symptoms while also being well-tolerated. For these reasons, Myqorzo represents an important step forward in how we care for people living with obstructive HCM.”

https://en.wikipedia.org/wiki/Aficamten

FDA Approves Myqorzo (aficamten) for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy